基因療法有助於治療X染色體有關的免疫缺陷綜合症狀

能消除感染,重建免疫系統的功能,使患者擁有正常的生活

  4月18日出版的《新英格蘭醫學雜誌》中發表的一項研究結果發現,基因治療對X染色體相關的重症聯合免疫缺陷患者有琠w的療效。在5名患者中,有4名患者的免疫功能得到了極大改善,從而擁有了正常的生活。

  巴黎INSERM實驗室的Salima Hacein-Bey-Abina博士及同事寫到:「X染色體相關的重症聯合免疫缺陷[SCID]的發病原因,是編碼γ鏈的基因發生突變。這是一種致死性疾病,能通過異體幹細胞移植得到治癒。將自身造血幹細胞中編碼γ鏈的基因進行在體外進行轉換後,再輸入患者體內,我們調查了這種方法是否能重建這些患者的免疫系統。」

  調查者使用一種有缺陷的逆轉錄病毒載體,把5名重症聯合免疫缺陷患兒的CD34+骨髓細胞取出體外並進行轉換。這種做法對患者無任何不良影響。5名患兒中的4名,在4個月內,血液中出現轉換後的T細胞和自然殺傷細胞,免疫功能的改善一直持續到2年後。儘管出現轉換後B細胞的頻率較低,但是血清免疫球蛋白水平和抗體的產量,足以使患者無須靜脈輸注免疫球蛋白(IVIG)。

  作者寫到:「免疫缺陷的糾正消除了確定的感染,使患者能擁有正常的生活。」

  在一篇附論中,波士頓血液病研究中心的Fred S. Rosen醫師建議,X染色體相關的重症聯合免疫缺陷患者的骨髓幹細胞中插入轉基因,在骨髓移植後可能會重建功能性B細胞。他寫到:「避免靜脈輸注免疫球蛋白治療,會導致『巨大的經濟利益和生活方式的改進。』」

  

Gene Therapy Helpful in X-Linked Immunodeficiency Syndrome

Can Eradicate Infection, Restore Immune System, Allow for Normal Life

By Laurie Barclay, MD
WebMD Medical News

Reviewed by Gary D. Vogin, MD

April 17, 2002 -- Gene therapy has sustained benefit for patients with a particular form of X-linked severe combined immunodeficiency syndrome, according to results of a study published in the April 18 issue of The New England Journal of Medicine. Four out of five patients had sufficient improvement in their immune function to lead normal lives.

"X-linked severe combined immunodeficiency syndrome [SCID] due to a mutation in the gene encoding the common gamma (gamma-c) chain is a lethal condition that can be cured by allogeneic stem-cell transplantation," write Salima Hacein-Bey-Abina, PhD, from the Laboratoire INSERM in Paris, and colleagues. "We investigated whether infusion of autologous hematopoetic stem cells that had been transduced in vitro with the gamma-C gene can restore the immune system in [these] patients."

Using a defective retroviral vector, the investigators transduced ex vivo CD34+ bone marrow cells from five boys with this SCID, resulting in no adverse effects. In four of the five boys, transduced T cells and natural killer cells appeared in the blood within four months, and improvements in immune function were sustained up to two years. Despite the low frequency of transduced B cells, serum immunoglobulin levels and antibody production were sufficient to avoid the need for intravenous immunoglobulin (IVIG).

"Correction of the immunodeficiency eradicated established infections and allowed patients to have a normal life," the authors write.

In an accompanying editorial, Fred S. Rosen, MD, from the Center for Blood Research in Boston, suggests that inserting the transgene into the bone marrow stem cells of patients with X-linked SCID might establish functional B cells after bone marrow transplant. Avoiding IVIG therapy would result in "enormous economic and lifestyle benefits," he writes.

© 2002 WebMD Inc. All rights reserved.

    
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